Is oxytocin a safe treatment for autism?

“Got Questions?” is a new weekly feature on our blog to address the desire for scientific understanding in our community. We received over 3000 responses when we asked what science questions were on your mind. We answered a few here and the Autism Speaks Science staff will address the other themes we received in this weekly post.

The hormone oxytocin is believed to play a role in social bonding and affiliation. Researchers using animal studies have shown that oxytocin released in the brain regulates social recognition, social memory, mother-infant and male-female bonding, and other aspects of attachment. Because social impairments are one of its most consistent features of autism, a number of scientists have hypothesized that abnormal oxytocin function might play a role in autism spectrum disorders (ASD).

The data regarding whether individuals with ASD have changes in oxytocin signaling are somewhat mixed. A few studies have found children with ASD have lower average levels of blood oxytocin in comparison to typically developing children matched for age. Several genetic studies have found associations between autism and specific forms of the gene for the oxytocin receptor. Recently, it was reported that post-mortem brain tissue from individuals with ASD showed lower levels of gene expression for the oxytocin receptor when compared to age/gender matched comparison individuals.

So far only a few studies have examined the effect of administering oxytocin in human beings. The data suggest that administration of oxytocin, either nasally or intravenously, positively impacts human social behavior. For instance, recent studies with typical individuals show that giving oxytocin can increase gaze to the eye region of human faces, increase memory for faces, and improve the ability of people to infer the mental states of others (known as Theory of Mind). All of these are challenges that have been associated with autism.

Given the lack of pharmacologic treatments for social deficits and the possibility of dysregulated oxytocin signaling in autism, many have proposed oxytocin as a treatment for ASD. These studies are just beginning. The three reports published so far have suggested that a single dose of oxytocin can temporarily improve social cognition in adults and, in one study, teenagers, with ASD. In a pilot placebo-controlled study, researchers found that twice daily oxytocin in adults with autism resulted in some improvements in social function and quality of life and reductions in certain types of repetitive behaviorsThe data are very preliminary and larger follow-up studies are now underway.

Together these first studies support a potential role of oxytocin in ASD and suggest oxytocin could have therapeutic benefits for the treatment of ASD symptoms in adults, especially in the area of social functioning. It is important to note that there are currently no published safety or efficacy data for oxytocin in children.

Autism Speaks is funding research to better understanding the role of oxytocin in ASD. More information on clinical trials for oxytocin as a treatment for ASD can be found at www.clinicaltrials.gov.